The Food and Drug Administration approved Novartis’s gene therapy drug that treats a rare and deadly disease affecting children. Zolgensma (onasemnogene abeparvovec-xioxi) was approved for children under the age of 2 with spinal muscular atrophy to use. Novartis gene therapy is the most expensive drug ever to be approved and marketed, a price tag that shatters Sparks Therapeutics’ gene therapy drug meant to repair vision impairment caused by a rare disease. Spark therapeutics’ drug held the record at first when it price its gene therapy at $425,000 per eye, running you up to $850,000 if you wanted to return vision on both eyes.
The CEO of Novartis was going to originally price Zolgensma at $5 million but decided that a price of $2 million was a more conservative price. Novartis broke a record in pharmaceutical drug pricing by making its gene therapy cost $2.125 million per patient.
How does Zolgensma work with SMA?
Spinal muscular atrophy (SMA) is a terrible disease that slowly deteriorates a child’s muscle if left untreated. It is estimated every year that 200,000 children in the United States are affected by a genetic mutation resulting to SMA. The mutation lies on the telomeric and centromeric halves on chromosome 5q13. The loss of SMN1 Survival motor neuron (SMN1) or component of gems 1 (GEMIN1) contains the Sequence needed to produce SMN proteins. Without SMN1, children can’t make the protein it needs for their muscles to develop normally.
Before AveXis, children with spinal muscular atrophy would be lucky enough to live past their 2nd birthday. Zolgensma (onasemnogene abeparvovec-xioi) is meant to treat children as soon as possible to catch the disease early. Zolgensma replaces the SMN1 gene that is defective or missing with a healthier and more functional version of the gene.
Why is the Drug so Expensive?
Drugs are always expensive when you consider the risk and the investment it takes to bring a drug to the market ( which you hope will end up working out in the end). Even if you invest in creating the drug, there is no guarantee that the drug will work.
Gene therapy isn’t a new field, it was once believed that the science in this field was too new and early to make a difference in medicine. Boy, we were wrong. Novartis’s team of scientists engineered Zolgensma to effectively cure spinal muscle atrophy. A feat that shouldn’t be left unnoticed or unheard, spinal muscle atrophy was the number one mortality for children suffering from genetic mutations.
A terrible disease that slowly kills children by deteriorating their muscles but now children like Donovan Weisgarber will live past their 2nd birthday.
Gene therapy drugs are pricier, but we shouldn’t be surprise. The benefits and quality of life that Zolgensma returns to children with SMN1 mutation is outstanding. A cure to a fatal disease, expensive but reasonable when you look at how many birthdays children like Donovan will have after their therapy.